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Hemophilia B Topic Center

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DTX101 Gene Therapy Ends Development, Offers Key Lessons for Future Hemophilia B Treatment Strategies
09/16/2025
Grace Taylor, MS, MA
Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
Early-phase trials of the DTX101...
09/16/2025
First Report Managed Care
News
Study Confirms Long-Term Safety and Efficacy of AAV Gene Therapy in Hemophilia B
07/22/2025
Grace Taylor, MS, MA
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A 13-year follow-up study shows...
07/22/2025
First Report Managed Care
News
Community Counts Study Identifies Predictors of Joint Bleeding in Severe Hemophilia
06/12/2025
Grace Taylor, MS, MA
A Community Counts Longitudinal Surveillance Cohort study reveals that modern therapies, treatment adherence, and social factors all play pivotal roles in reducing joint bleeding in severe hemophilia.
A Community Counts Longitudinal Surveillance Cohort study reveals that modern therapies, treatment adherence, and social factors all play pivotal roles in reducing joint bleeding in severe hemophilia.
A Community Counts Longitudinal...
06/12/2025
First Report Managed Care
News
Fitusiran Shows Promise as Long-Term Bleed Prevention Therapy in Hemophilia A and B
06/04/2025
Grace Taylor, MS, MA
The phase 3 ATLAS-OLE study shows that fitusiran may offer long-lasting bleed protection for people with hemophilia A or B.
The phase 3 ATLAS-OLE study shows that fitusiran may offer long-lasting bleed protection for people with hemophilia A or B.
The phase 3 ATLAS-OLE study...
06/04/2025
First Report Managed Care
News
One-Time Gene Therapy Achieves Long-Term Bleeding Control in Hemophilia B
05/14/2025
Grace Taylor, MS, MA
Researchers report that a single-dose gene therapy led to sustained clotting factor levels and significant reductions in bleeding and treatment burden for patients with hemophilia B.
Researchers report that a single-dose gene therapy led to sustained clotting factor levels and significant reductions in bleeding and treatment burden for patients with hemophilia B.
Researchers report that a...
05/14/2025
First Report Managed Care
News
Emicizumab as a Potential Treatment Pathway for Select Hemophilia B Cases
11/22/2024
Lisa Kuhns, PhD, MD
New research highlights the potential of emicizumab, traditionally used to treat hemophilia A (HA), as a promising alternative therapy for certain hemophilia B (HB) genotypes.
New research highlights the potential of emicizumab, traditionally used to treat hemophilia A (HA), as a promising alternative therapy for certain hemophilia B (HB) genotypes.
New research highlights the...
11/22/2024
First Report Managed Care
News
Revolutionary Single-Dose Gene Therapy Offers New Hope for Adults with Hemophilia B
11/08/2024
Lisa Kuhns, PhD, MD
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy...
11/08/2024
First Report Managed Care
News
Advances in Factor IX Therapy Offer New Hope for Hemophilia B Treatment
10/23/2024
Lisa Kuhns, PhD, MD
New factor IX therapies promise improved treatment for hemophilia B.
New factor IX therapies promise improved treatment for hemophilia B.
New factor IX therapies promise...
10/23/2024
First Report Managed Care
News
New Guidance Addresses Management of Patients With Congenital Hemophilia
10/09/2024
Jolynn Tumolo
New guidelines from the International Society on Thrombosis and Haemostasis strongly recommend prophylactic treatment over episodic for patients with severe and moderately severe hemophilia A and B, with conditional recommendations for other...
New guidelines from the International Society on Thrombosis and Haemostasis strongly recommend prophylactic treatment over episodic for patients with severe and moderately severe hemophilia A and B, with conditional recommendations for other...
New guidelines from the...
10/09/2024
First Report Managed Care
News
New 4F-PCC Proves Effective in Rapid Reversal of Anticoagulation for Urgent Surgery, Clinical Trial Finds
09/10/2024
Lisa Kuhns, PhD, MD
An investigational 4-factor prothrombin complex concentrate (4F-PCC) is as effective and safe as an FDA-approved 4F-PCC for rapidly reversing vitamin K antagonist (VKA) anticoagulation in patients requiring urgent surgery, according to a...
An investigational 4-factor prothrombin complex concentrate (4F-PCC) is as effective and safe as an FDA-approved 4F-PCC for rapidly reversing vitamin K antagonist (VKA) anticoagulation in patients requiring urgent surgery, according to a...
An investigational 4-factor...
09/10/2024
First Report Managed Care
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